Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model

Nature Communications

Volumn 6, Issue , 2015, Pages

  Bissig Choisat, Beatrice ^a   Wang, Lili ^b   Legras, Xavier ^a   Saha, Pradip K ^c   Chen, Leon ^a   Bell, Peter ^b   Pankowicz, Francis P ^a,c   Hill, Matthew C ^a,c   Barzi, Mercedes ^a   Leyton, Claudia Kettlun ^a   Leung, Hon Chiu Eastwood ^c,d   Kruse, Robert L ^a,c   Himes, Ryan W ^e   Goss, John A ^e   Wilson, James M ^b   Chan, Lawrence ^c   Lagor, William R ^c   Bissig, Karl Dimiter ^a,c  

^a CENTER FOR CELL AND GENE THERAPY   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c BAYLOR COLLEGE OF MEDICINE   (United States)

^d Texas Children's Hospital ^*  (United States)

^e TEXAS CHILDREN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

APOLIPOPROTEIN A1; CHOLESTEROL ESTER TRANSFER PROTEIN; HIGH DENSITY LIPOPROTEIN; LOW DENSITY LIPOPROTEIN RECEPTOR; LDLR PROTEIN, HUMAN; LIPOPROTEIN;

BLOOD; DRUG; GENE TRANSFER; HOMINID; LIPID; METABOLISM; MORBIDITY; PROTEIN; RODENT; SERUM;

ADENO ASSOCIATED VIRUS 9; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CHILD; CHOLESTEROL BLOOD LEVEL; CONTROLLED STUDY; FAMILIAL HYPERCHOLESTEROLEMIA; GENE EXPRESSION; GENE THERAPY; HEPATOCYTE TRANSPLANTATION; HUMAN; HUMAN CELL; LIPOPROTEIN METABOLISM; LIVER CELL; LIVER TRANSPLANTATION; LOSS OF FUNCTION MUTATION; MOUSE; NONHUMAN; SCHOOL CHILD; XENOGRAFT; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; FEMALE; GENETICS; HYPERLIPOPROTEINEMIA TYPE II; METABOLISM; TRANSPLANTATION;

ADENO-ASSOCIATED VIRUS 9; ANIMALIA; MUS;

ANIMALS; CHILD; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FEMALE; GENETIC THERAPY; HEPATOCYTES; HETEROGRAFTS; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; LIPOPROTEINS; RECEPTORS, LDL;

EID: 84935897836     PISSN: None     EISSN: 20411723     Source Type: Journal    
DOI: 10.1038/ncomms8339     Document Type: Article

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